Regulatory Authority Actions

With this approval, HYQVIA [Immune Globulin Infusion 10% (human) with Recombinant Human Hyaluronidase] becomes the first and only facilitated subcutaneous immunoglobulin available in Japan to treat these disorders.

The comment period for the Federal Register Notice, “Evaluating the Immunogenicity Risk of Host Cell Proteins in Follow-on Recombinant Peptide Products: Establishment of a Public Docket: Request for Information and Comments”, has been reopened by FDA until March 3, 2025.

FDA's approval of Hikma Pharmaceuticals’ liraglutide injection makes this product the first generic version of Victoza, a GLP-1 receptor agonist.

The Center for Drug Evaluation and Research’s new center will coordinate and promote use of real-word data in regulatory decisions.

FDA's approval will allow Kedrion to manufacture Ryplazim (plasminogen, human-tvmh), the only FDA-approved therapy for treating PLGD-1, at facility in Bolognana, Italy.

FDA's approval of eladocagene exuparvovec-tneq (Kebilidi) makes that product the first FDA-approved gene therapy for treating AADC deficiency.

CHMP has granted Eisai Europe and Biogen a positive opinion for the use of their monoclonal antibody therapy in treating early Alzheimer’s disease.

Balversa (erdafitinib) marks the first and only bladder cancer therapy to target FGFR3 alterations, with a demonstrated increase in overall survival from 7.8 months to 12.1 months.

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post.

Vyalev, also known as Produodopa, has been approved in 35 countries, with more than 4200 patients having started treatment.

As many as 30% of people living with severe hemophilia A develop inhibitors such as TFPI, which can be produced by the body’s immune response to clotting factors in replacement therapy, often limiting the effectiveness of that therapy.

Wainzua had been previously approved for use by patients with ATTRv-PN in the United States, under the brand name Wainua.

The new mechanism of action targets cholinergic receptors instead of the standard dopamine receptors.

The agency is recommending that the market authorization for Oxbryta (voxelotor) be suspended so that emerging data may be reviewed.

Bio/pharma companies should be approaching their decarbonization journey as a strategic business move for the future as industry stakeholders are placing greater value on sustainability.

Phase III trial results showed that Imfinzi (durvalumab in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery.

PiaSky (crovalimab) has previously been approved in countries such as the United States and Japan for monthly, subcutaneous use that may be self-administered with adequate training.

Opaganib was given orphan drug designation for the treatment of neuroblastoma in children, which is rare, but the most common infancy malignancy.

In a 24-week study of patients with PAH, six-minute walking distance improved by an average of 40.8 meters, and risk of death or clinical worsening was reduced by 82%.

The combination of Opdivo plus Yervoy is already established as a second-line treatment for patients with advanced hepatocellular carcinoma.

The International Coalition of Medicines Regulatory Authorities’ new Collaborative Hybrid Inspection Pilot program allows a CDMO to gain approval from multiple countries simultaneously, as opposed to separately phased inspections from different corners of the globe.

Fasenra, which has been approved since 2019 in the United States in a pre-filled, single-use auto-injector form that can be administered either in a doctor’s office or at home, is now approved in China for age 12 and up.

Approximately 15% of all lung cancers diagnosed in the US are of the small-cell variety, and of those, 70% are considered extensive-stage.

NexoBrid, a topically administered biological product, is now authorized for use in all age groups in the United States, European Union, and Japan.

Breyanzi demonstrated an overall high response rate in adults with relapsed or refractory follicular lymphoma in a Phase II study.

Imfinzi may be used alone, or with chemotherapy in tandem with the other approved treatment, Lynparza.

Yorvipath is a prodrug of parathyroid hormone (PTH) (1-34), designed to be administered once daily to provide continuous exposure to released PTH.

AstraZeneca and Daiichi Sankyo first entered into an agreement to develop Enhertu in March 2019, and the antibody-drug conjugate has since been approved for various groups in more than 65 countries.

The epinephrine nasal spray was approved for the emergency treatment of type 1 allergic reactions in adults and children.

The reopening of comments addresses the addition of a warning to consumers that acetaminophen may cause skin irritation characterized by reddening, blisters, and rashes.